Hepatocelluar carcinoma (HCC), of which the prevalence in Asia Pacific area including Taiwan is high and is an important cause of death (El-Serag H B, Rudolph K L. Hepatocellular carcinoma: epidemiology and molecular carcinogenesis. Gastroenterology 2007; 132: 2557-2576). Surgical resection remains the way most promisingly leading to long-term survival; however, only 20% or less of the patients with HCC could undertake surgical resection (Hung H. Treatment modalities for hepatocellular carcinoma. Curr Cancer Drug Targets 2005; 5: 131-138.). There is still an unmet need for systemic therapies for advanced HCC (Lopez P M, Villanueva A, Llovet J M. Systematic review: evidence based management of hepatocellular carcinoma—an updated analysis of randomized controlled trials. Aliment Pharmacol Ther 2006; 23: 1535-1547) even after sorafenib was proven to improve overall survival (OS) in patients with advanced HCC (Llovet J M, Ricci S, Mazzaferro V, et al. Sorafenib in advanced hepatocellular carcinoma. N Engl J Med 2008; 359: 378-390). Sorafenib significantly prolonged OS of patients with advanced HCC; however, the increment was small especially for patients in Asia Pacific area where around 70% of HCC are HBV associated, with median OS of 6.5 months in the sorafenib arm compared with 4.2 months in the placebo arm (Cheng A L, Kang Y K, Chen Z, Tsao C J, Qin S, Kim J S, Luo R, Feng J, Ye S, Yang T S, Xu J, Sun Y, Liang H, Liu J, Wang J, Tak W Y, Pan H, Burock K, Zou J, Voliotis D, Guan Z. Efficacy and safety of sorafenib in patients in the Asia-Pacific region with advanced hepatocellular carcinoma: a phase III randomised, double-blind, placebo-controlled trial. Lancet Oncol. 2009 January; 10(1):25-34).
There is still a need for new systemic therapies for a cancer with synergistic efficacies.